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Now Recruiting

A Study of Intrathecal SHP611 in Participants with Late Infantile Metachromatic Leukodystrophy

WHAT IS THE PURPOSE OF THIS STUDY?

The primary purpose of this study is to evaluate the effects of intrathecal (IT) administration of SHP611 on gross motor function, using the Gross Motor Function Classification in Metachromatic Leukodystrophy (GMFC-MLD) compared with matched historical control data in children with metachromatic leukodystrophy (MLD).

Study Identifiers

IS THIS STUDY RIGHT FOR ME?

AGE

6 to 72

Months

GENDERS

ACCEPTS HEALTHY VOLUNTEERS

No

PARTICIPATION DURATION/ REQUIREMENTS

It is planned to enroll approximately 42 participants in study SHP611-201, with a participant’s expected maximum duration of participation being approximately 26 months from screening to the last scheduled visit. The study will consist of a screening period of up to 28 days. All participants will receive a once weekly dose of intrathecal (IT) SHP611 at 150 mg for 105 weeks via an intrathecal drug delivery device (IDDD) that will be implanted during a period of up to 10 days before the first administration of IT SHP611. If use of the IDDD is not possible (particularly for younger participants), a lumbar puncture may be utilized. During treatment, participants will undergo assessments of gross motor function, brain imaging and quality of life. Assessment of gross motor function will be video recorded. X-rays may be performed as needed throughout the study (particularly for younger participants) to confirm placement of the device and/or evaluate a nonworking catheter. Participants who complete the study will be offered the opportunity to continue in a separate extension study (SHP611-202). For participants who wish to discontinue study SHP611-201, a safety follow-up visit will be conducted at Week 108 and the IDDD will be removed upon discontinuation of the study. If due to safety reasons the IDDD remains in the participant beyond the last study visit, the participant will continue to be observed for safety in an extension study at visits to the site every 6 months until the IDDD has been fully removed.

ENTRY CRITERIA

Must be >=12 to 72 months of age (or >=6 to <18 months of age at screening, if younger siblings of enrolled participants and have the same ASA allelic constitution)

Must have a documented diagnosis of MLD

Must have a gait disorder due to spastic ataxia or weakness attributed to MLD by 30 months of age or be minimally or early symptomatic

Must have a Gross Motor Function Classification in Metachromatic Leukodystrophy (GMFC-MLD) level 1 – 6 at screening or be minimally symptomatic

Cannot have a multiple sulfatase disorder as determined by abnormal activity of other lysosomal sulfatase or a known genetic disorder other than MLD

Cannot have a history of bone marrow transplant (BMT), hematopoietic stem cell transplantation (HSCT) or gene therapy or undergo HSCT or gene therapy at any time during the study

Primary presentation of MLD cannot have been behavioral or cognitive symptoms (per investigator’s clinical judgment); behavioral symptoms that are secondary to motor deficits (eg, tantrums in response to loss of motor skills) are not exclusionary

Cannot have any known or suspected hypersensitivity to agents used for anesthesia or have a history of difficult airway or potential for airway compromise

Cannot be enrolled in another clinical study involving the use of any investigational product (drug or device) within 30 days or 5 half-lives (whichever is longer) prior to study enrollment or at any time during the study

Cannot have a condition that is contraindicated to the intrathecal drug delivery device (IDDD) used in study SHP611-201

Study Locations

We are planning* on conducting this study in 9 countries around the world.

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Important information regarding the use of this website

You are about to download Clinical Study Report (CSR) synopses or factual lay summaries on clinical trials for medicines/indications approved in the US and EU on or after January 1, 2015.
Access to these documents requires that you acknowledge and accept the following:
• The purpose of this website is to increase transparency regarding the outcome of clinical trials sponsored by Shire and is strictly non-promotional. It is not intended to promote any prescription, sale or use, whether approved or off-label, of any Shire medicinal products. This website is for informational purposes only and is not intended to have any legally binding effect.
• Members of the public having any questions regarding the use of any of the products referred to in these documents should consult a healthcare professional for additional information and medical advice. They should not rely on the information from this website to draw any conclusions about healthcare products, treatments or treatment options.
• The information provided on this website is not intended to be used as prescribing information. Please refer to applicable authority-approved labeling information for your country.
• The documents on this website are provided in the same format in which they were originally prepared for submission to regulatory authorities for marketing approval; exceptions include redactions necessary for protection of personal data and commercially confidential information.
• The information contained in these documents represents the results known at the time of the study’s completion. Shire does not accept any liability for the accuracy, completeness or utility of this information. There is no obligation in connection with updating the documents and information available on this website. Your access and use of this website and the content it contains is at your own risk.

Important information regarding the use of this website

You are about to download Clinical Study Report (CSR) synopses or factual lay summaries on clinical trials for medicines/indications approved in the US and EU on or after January 1, 2015.
Access to these documents requires that you acknowledge and accept the following:
• The purpose of this website is to increase transparency regarding the outcome of clinical trials sponsored by Shire and is strictly non-promotional. It is not intended to promote any prescription, sale or use, whether approved or off-label, of any Shire medicinal products. This website is for informational purposes only and is not intended to have any legally binding effect.
• Members of the public having any questions regarding the use of any of the products referred to in these documents should consult a healthcare professional for additional information and medical advice. They should not rely on the information from this website to draw any conclusions about healthcare products, treatments or treatment options.
• The information provided on this website is not intended to be used as prescribing information. Please refer to applicable authority-approved labeling information for your country.
• The documents on this website are provided in the same format in which they were originally prepared for submission to regulatory authorities for marketing approval; exceptions include redactions necessary for protection of personal data and commercially confidential information.
• The information contained in these documents represents the results known at the time of the study’s completion. Shire does not accept any liability for the accuracy, completeness or utility of this information. There is no obligation in connection with updating the documents and information available on this website. Your access and use of this website and the content it contains is at your own risk.